Ontology highlight
ABSTRACT:
SUBMITTER: Gao QQ
PROVIDER: S-EPMC4639981 | biostudies-literature | 2015 Nov
REPOSITORIES: biostudies-literature
Gao Quan Q QQ Wyatt Eugene E Goldstein Jeff A JA LoPresti Peter P Castillo Lisa M LM Gazda Alec A Petrossian Natalie N Earley Judy U JU Hadhazy Michele M Barefield David Y DY Demonbreun Alexis R AR Bönnemann Carsten C Wolf Matthew M McNally Elizabeth M EM
The Journal of clinical investigation 20151012 11
Exon skipping uses antisense oligonucleotides as a treatment for genetic diseases. The antisense oligonucleotides used for exon skipping are designed to bypass premature stop codons in the target RNA and restore reading frame disruption. Exon skipping is currently being tested in humans with dystrophin gene mutations who have Duchenne muscular dystrophy. For Duchenne muscular dystrophy, the rationale for exon skipping derived from observations in patients with naturally occurring dystrophin gene ...[more]